Tag Archives: Rare Disease Drug Development

Mitochondrial Disease: The Most Common Disease You’ve Probably Never Heard of… About 20 years ago, mitochondrial disease was virtually unheard of. Just 10 years ago, it was considered rare. Today, according to NORD, a disease is considered rare when less than 200,000 people are affected. Mitochondrial disease is not a single disease, but rather an entire umbrella of rare diseases. For example, Alpers disease and Barth syndrome are very different and rare forms of mitochondrial Read More >

There are more than 6,000 different rare diseases affecting over 60 million people across Europe and North America, alone, and millions more throughout the world. Most of these diseases are genetic, serious, chronic and debilitating. Each disease is different, but they affect people in similar ways. Joining together can help patients and their families find common solutions for care and remind them they are not alone. As the world marks Rare Disease Day on February Read More >

One of the most important keys to successful relationships – be it in life or rare disease drug development – is open, honest and frequent communication, according to a panel of rare disease experts speaking at the Therapeutic Workshop: “Balancing the Rick-Reward in Rare Disease Drug Development” at the BIO CEO & Investor Conference in New York on Monday. Without engaging the key groups at the right time and the right manner, companies entering this Read More >